FDA approves first combo treatment for BRAF-positive anaplastic thyroid cancer

May 7, 2018

A combination cancer treatment has been expanded to include treating both melanoma and anaplastic thyroid cancer (ATC).

A combination cancer treatment has been expanded to include treating both melanoma and anaplastic thyroid cancer (ATC).

FDA recently approved Novartis’s dabrafenib (Tafinlar) and trametinib (Mekinist), administered together, for the treatment of metastatic ATC, which has a type of abnormal gene, BRAF V600E (BRAF V600E mutation-positive). The combo drug was also approved for patients with melanoma, with BRAF V600E or V600K mutations and involvement of lymph node(s), following complete resection.

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“This is the first FDA-approved treatment for patients with this aggressive form of thyroid cancer, and the third cancer with this specific gene mutation that this drug combination has been approved to treat,” said Richard Pazdur, MD, director of FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research, in an FDA statement. “This approval demonstrates that targeting the same molecular pathway in diverse diseases is an effective way to expedite the development of treatments that may help more patients.”

"Since the initial approval of Tafinlar and Mekinist in metastatic melanoma in 2013, the combination has become an important therapy for many patients carrying a BRAF mutation in both melanoma and lung cancers," said Liz Barrett, CEO of Novartis Oncology, in a Novartis statement.

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The efficacy of Tafinlar and Mekinist in treating ATC was shown in an open-label clinical trial of patients with rare cancers with the BRAF V600E mutation. The trial measured the percent of patients with a complete or partial reduction in tumor size (overall response rate). Of 23 evaluable patients, 57% experienced a partial response and 4% experienced a complete response. In 64% of the 14 patients with responses, there were no significant tumor growths for six months or longer.

Meanwhile, the melanoma approval is based on results from a phase 3 study of 870 patients with Stage 3 BRAF V600E/K mutation-positive melanoma treated with Tafinlar and Mekinist after complete surgical resection. Treatment with the combination therapy significantly reduced the risk of disease recurrence or death by 53%.

Improvements were also observed in key secondary end points including overall survival, distant metastasis-free survival and freedom from relapse.

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